Abstract 3725: Simvastatin but Not Pravastatin Affects Sleep: Findings from the UCSD Statin Study

Circulation ◽  
2007 ◽  
Vol 116 (suppl_16) ◽  
Author(s):  
Beatrice A Golomb ◽  
Edwin K Kwon ◽  
Michael H Criqui ◽  
Joel E Dimsdale
Keyword(s):  
Sleep Quality ◽  
Rating Scale ◽  
Sleep Problems ◽  
Small Sample Size ◽  
Case Reports ◽  
Controlled Trial ◽  
Small Sample ◽  
T Test ◽  
Secondary Outcome ◽  
Regression Analyses

Background : Case reports have suggested possible effects of lipophilic statins on sleep in some subjects. Most randomized studies evaluating the effect of statins on sleep have had small sample size and short duration (≤ 6 weeks). Whether statins affect sleep on average, favorably or adversely, has been unclear. Goal : To assess the effects of lipophilic and hydrophilic statins on sleep. Subjects : 1016 adult men and women without diabetes or heart disease, with LDL-cholesterol 115–190mg/dL. Design : Randomized double blind placebo-controlled trial of simvastatin 20mg, pravastatin 40mg or placebo for 6 months. Sleep was a prespecified secondary outcome. It was assessed by both an adaptation of the Leeds sleep scale (a visual analog scale of sleep quality); and a rating scale of sleep problems. Both items were measured at baseline and on-treatment. Analysis : Baseline comparability of randomization groups including sleep measures was affirmed. T-test of mean on-treatment sleep scores across randomization groups was performed. This complemented regression analyses, adjusted for baseline values of the respective sleep assessment. Results : Groups were comparable at baseline on variables including both sleep measures. Simvastatin use was associated with significantly worse sleep quality, and significantly greater reported sleep problems than either pravastatin or placebo, by t-test and regression analyses. Pravastatin did not differ significantly from placebo on any sleep outcome. Conclusion : Findings were compatible with the hypothesis that statins may impair sleep in some subjects, and that this impairment may arise selectively with lipophilic statins. Table 1. Effects of Statins on Sleep: Regression Analysis

A Pilot Randomized Sham-Controlled Trial of MC5-A Scrambler Therapy in the Treatment of Chronic Chemotherapy-Induced Peripheral Neuropathy (CIPN)

2019 ◽  
Vol 35 (1) ◽  
pp. 53-58 ◽  
Author(s):  
Thomas J. Smith ◽  
A. Rab Razzak ◽  
Amanda L. Blackford ◽  
Jennifer Ensminger ◽  
Catherine Saiki ◽  
...  
Keyword(s):  
Peripheral Neuropathy ◽  
Rating Scale ◽  
Small Sample Size ◽  
Controlled Trial ◽  
Brief Pain Inventory ◽  
Small Sample ◽  
Sham Treatment ◽  
The Real ◽  
Average Pain ◽  
Scrambler Therapy

Background: Chemotherapy-induced peripheral neuropathy (CIPN) affects 30% to 40% of patients with cancer with long-lasting disability. Scrambler therapy (ST) appeared to benefit patients in uncontrolled trials, so we performed a randomized sham-controlled Phase II trial of ST. Methods: The primary end point was “average pain” after 28 days on the Numeric Rating Scale. Each received ten 30-minute sessions of ST on the dermatomes above the painful areas, or sham treatment on the back, typically at L3-5 where the nerve roots would enter the spinal cord. Outcomes included the Brief Pain Inventory (BPI)-CIPN and the EORTC CIPN-20 scale. Patients were evaluated before treatment (day 0), day 10, and days 28, 60, and 90. Results: Data regarding pain as a primary outcome were collected for 33 of the 35 patients. There were no significant differences between the sham and the “real” ST group at day 10, 28, 60, or 90, for average pain, the BPI, or EORTC CIPN-20. Individual responses were noted during the ST treatment on the real arm, but most dissipated by day 30. There was improvement in the sensory subscale of the CIPN-20 at 2 months in the “real” group ( P = .14). All “real” patients wanted to continue treatment if available. Discussion: We observed no difference between sham and real ST CIPN treatment. Potential reasons include at least the following: ST does not work; the sham treatment had some effect; small sample size with heterogeneous patients; misplaced electrodes on an area of nonpainful but damaged nerves; or a combination of these factors.

scholarly journals Cisternostomy in Traumatic Brain Injury for a Novel Approach to Treatment: Review of Current Status

2021 ◽  
Author(s):  
Manish Agrawal ◽  
Keshav Mishra ◽  
Rohit Babal ◽  
Devendra Purohit
Keyword(s):  
Traumatic Brain Injury ◽  
Brain Injury ◽  
Clinical Outcome ◽  
English Language ◽  
Small Sample Size ◽  
Case Reports ◽  
Controlled Trial ◽  
Small Sample ◽  
Current Status ◽  
Level Of Evidence

AbstractCisternostomy (CS) is a recently introduced procedure to mitigate the cerebrospinal fluid shift edema and secondary injury in traumatic brain injury patients. There have been numerous reports describing its efficacy in various cohorts of traumatic brain injury (TBI) patients; therefore, we review the current evidences examining its utility for the same. The systematic review was done according to Preferred Reporting Items for Systematic Reviews and Meta-Analyses guideline including all human studies published in English language indexed with MEDLINE and Google Scholar since 2013 evaluating CS as a standalone or as an adjuvant procedure. The studies were graded according to the Oxford center for evidence-based medicine levels of evidence. Case reports were excluded from the analysis. A total of 107 articles were found by using the given search criteria. Finally, three oxford level 3 study and one level 4 study were included in the analysis. CS in combination with decompressive craniectomy was seen to confer mortality benefit and better outcome at discharge. It led to statistically significant improvement in clinical outcome at 6 months in the patients for whom it was performed as a primary procedure. CS is a promising procedure in TBI leading to survival benefit as well as better clinical outcome. But the level of evidence supporting its effectiveness is still weak owing to the methodological limitations and small sample size. A well-designed multicentric randomized controlled trial is needed to critically examine its role in TBI patients.

Comprehensive Geriatric Care to Improve Mobility after Hip Fracture: An RCT

Gerontology ◽  
2020 ◽  
Vol 66 (6) ◽  
pp. 542-548
Author(s):  
Wendy L. Cook ◽  
Penelope M.A. Brasher ◽  
Pierre Guy ◽  
Stirling Bryan ◽  
Meghan G. Donaldson ◽  
...  
Keyword(s):  
Hip Fracture ◽  
Small Sample Size ◽  
Controlled Trial ◽  
Geriatric Care ◽  
Small Sample ◽  
Community Dwelling ◽  
Post Discharge ◽  
Mobility Performance ◽  
Discharge Care ◽  
Comprehensive Geriatric Care

<b><i>Background:</i></b> Comprehensive geriatric care (CGC) for older adults during hospitalization for hip fracture can improve mobility, but it is unclear whether CGC delivered after a return to community living improves mobility compared with usual post-discharge care. <b><i>Objective:</i></b> To determine if an outpatient clinic-based CGC regime in the first year after hip fracture improved mobility performance at 12 months. <b><i>Methods:</i></b> A two-arm, 1:1 parallel group, pragmatic, single-blind, single-center, randomized controlled trial at 3 hospitals in Vancouver, BC, Canada. Participants were community-dwelling adults, aged ≥65 years, with a hip fracture in the previous 3–12 months, who had no dementia and walked ≥10 m before the fracture occurred. Target enrollment was 130 participants. Clinic-based CGC was delivered by a geriatrician, physiotherapist, and occupational therapist. Primary outcome was the Short Physical Performance Battery (SPPB; 0–12) at 12 months. <b><i>Results:</i></b> We randomized 53/313 eligible participants with a mean (SD) age of 79.7 (7.9) years to intervention (<i>n</i> = 26) and usual care (UC, <i>n</i> = 27), and 49/53 (92%) completed the study. Mean 12-month (SD) SPPB scores in the intervention and UC groups were 9.08 (3.03) and 8.24 (2.44). The between-group difference was 0.9 (95% CI –0.3 to 2.0, <i>p</i> = 0.13). Adverse events were similar in the 2 groups. <b><i>Conclusion:</i></b> The small sample size of less than half our recruitment target precludes definitive conclusions about the effect of our intervention. However, our results are consistent with similar studies on this population and intervention.

scholarly journals DIAGNOSIS OF ENDOCRINE DISEASE: The diagnostic performance of adrenal biopsy: a systematic review and meta-analysis

2016 ◽  
Vol 175 (2) ◽  
pp. R65-R80 ◽  
Author(s):  
Irina Bancos ◽  
Shrikant Tamhane ◽  
Muhammad Shah ◽  
Danae A Delivanis ◽  
Fares Alahdab ◽  
...  
Keyword(s):  
Systematic Review ◽  
Diagnostic Performance ◽  
Small Sample Size ◽  
Controlled Trial ◽  
Meta Analysis ◽  
Small Sample ◽  
Cochrane Central Register ◽  
Moderate Risk ◽  
Adrenal Biopsy ◽  
Adrenocortical Carcinomas

ObjectiveTo perform a systematic review of published literature on adrenal biopsy and to assess its performance in diagnosing adrenal malignancy.MethodsMedline In-Process and Other Non-Indexed Citations, MEDLINE, EMBASE, and Cochrane Central Register of Controlled Trial were searched from inception to February 2016. Reviewers extracted data and assessed methodological quality in duplicate.ResultsWe included 32 observational studies reporting on 2174 patients (39.4% women, mean age 59.8 years) undergoing 2190 adrenal mass biopsy procedures. Pathology was described in 1621/2190 adrenal lesions (689 metastases, 68 adrenocortical carcinomas, 64 other malignancies, 464 adenomas, 226 other benign, 36 pheochromocytomas, and 74 others). The pooled non-diagnostic rate (30 studies, 2013 adrenal biopsies) was 8.7% (95%CI: 6–11%). The pooled complication rate (25 studies, 1339 biopsies) was 2.5% (95%CI: 1.5–3.4%). Studies were at a moderate risk for bias. Most limitations related to patient selection, assessment of outcome, and adequacy of follow-up. Only eight studies (240 patients) could be included in the diagnostic performance analysis with a sensitivity and specificity of 87 and 100% for malignancy, 70 and 98% for adrenocortical carcinoma, and 87 and 96% for metastasis respectively.ConclusionsEvidence based on small sample size and moderate risk of bias suggests that adrenal biopsy appears to be most useful in the diagnosis of adrenal metastasis in patients with a history of extra-adrenal malignancy. Adrenal biopsy should only be performed if the expected findings are likely to alter the management of the individual patient and after biochemical exclusion of catecholamine-producing tumors to help prevent potentially life-threatening complications.

scholarly journals Sleep Quality and Levodopa Intestinal Gel Infusion in Parkinson’s Disease: A Pilot Study

2018 ◽  
Vol 2018 ◽  
pp. 1-6
Author(s):  
Oriol De Fabregues ◽  
Alex Ferré ◽  
Odile Romero ◽  
Manuel Quintana ◽  
José Álvarez-Sabin
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Pilot Study ◽  
Sleep Quality ◽  
Rem Sleep ◽  
Rating Scale ◽  
Sleep Problems ◽  
Periodic Leg Movements ◽  
Before And After ◽  
Hamilton Anxiety Rating Scale

Background. Sleep problems in patients with advanced Parkinson’s disease (PD) have a deleterious impact on quality of life. Objective. To assess the effect of levodopa-carbidopa intestinal gel (LCIG) infusion on sleep quality in advanced PD patients. Methods. Seven patients participated in a prospective pilot study. Before and after 6 months of LCIG infusion, an overnight polysomnography was performed and the Epworth Sleepiness Scale, fatigue scale, Pittsburgh Sleep Quality Index, Beck Depression Inventory, and the Hamilton Anxiety Rating Scale were administered. Results. PSG showed low sleep efficiency. REM sleep without atony was found in 5 patients. After 6 months of LCIG infusion, the percentage of REM sleep decreased as well as the number of arousals especially due to reduction of spontaneous arousals and periodic leg movements during REM sleep, but differences were not statistically significant. Also, scores of all study questionnaires showed a tendency to improve. Conclusion. The results show a trend toward an improvement of sleep quality after 6 months of LCIG infusion, although differences as compared to pretreatment values were not statistically significant. The sleep architecture was not modified by LCIG. Further studies with larger study samples are needed to confirm these preliminary findings.

scholarly journals The Add-On Effect of Lactobacillus plantarum PS128 in Patients With Parkinson's Disease: A Pilot Study

2021 ◽  
Vol 8 ◽  
Author(s):  
Chin-Song Lu ◽  
Hsiu-Chen Chang ◽  
Yi-Hsin Weng ◽  
Chiung-Chu Chen ◽  
Yi-Shan Kuo ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Lactobacillus Plantarum ◽  
Outcome Measures ◽  
Rating Scale ◽  
Controlled Trial ◽  
Secondary Outcome ◽  
Motor Deficits ◽  
Motor Symptoms ◽  
Non Motor Symptoms

Background:Lactobacillus plantarum PS128 (PS128) is a specific probiotic, known as a psychobiotic, which has been demonstrated to alleviate motor deficits and inhibit neurodegenerative processes in Parkinson's disease (PD)-model mice. We hypothesize that it may also be beneficial to patients with PD based on the possible mechanism via the microbiome-gut-brain axis.Methods: This is an open-label, single-arm, baseline-controlled trial. The eligible participants were scheduled to take 60 billion colony-forming units of PS128 once per night for 12 weeks. Clinical assessments were conducted using the Unified Parkinson's Disease Rating Scale (UPDRS), modified Hoehn and Yahr scale, and change in patient “ON-OFF” diary recording as primary outcome measures. The non-motor symptoms questionnaire, Beck depression inventory-II, patient assessment of constipation symptom, 39-item Parkinson's Disease Questionnaire (PDQ-39), and Patient Global Impression of Change (PGI-C) were assessed as secondary outcome measures.Results: Twenty-five eligible patients (32% women) completed the study. The mean age was 61.84 ± 5.74 years (range, 52–72), mean disease duration was 10.12 ± 2.3 years (range, 5–14), and levodopa equivalent daily dosage was 1063.4 ± 209.5 mg/daily (range, 675–1,560). All patients remained on the same dosage of anti-parkinsonian and other drugs throughout the study. After 12 weeks of PS128 supplementation, the UPDRS motor scores improved significantly in both the OFF and ON states (p = 0.004 and p = 0.007, respectively). In addition, PS128 intervention significantly improved the duration of the ON period and OFF period as well as PDQ-39 values. However, no obvious effect of PS128 on non-motor symptoms of patients with PD was observed. Notably, the PGI-C scores improved in 17 patients (68%). PS128 intervention was also found to significantly reduce plasma myeloperoxidase and urine creatinine levels.Conclusion: The present study demonstrated that PS128 supplementation for 12 weeks with constant anti-parkinsonian medication improved the UPDRS motor score and quality of life of PD patients. We suggest that PS128 could serve as a therapeutic adjuvant for the treatment of PD. In the future, placebo-controlled studies are needed to further support the efficacy of PS128 supplementation.Clinical Trial Registration:https://clinicaltrials.gov/, identifier: NCT04389762.

scholarly journals Comparison of statistical methods for analysis of small sample sizes for detecting the differences in efficacy between treatments for knee osteoarthritis

2020 ◽  
Author(s):  
Chia-Lung Shih ◽  
Te-Yu Hung
Keyword(s):  
Knee Osteoarthritis ◽  
Sample Size ◽  
Statistical Methods ◽  
Statistical Power ◽  
Permutation Test ◽  
Small Sample Size ◽  
Small Sample ◽  
T Test ◽  
Sample Sizes ◽  
Knee Oa

Abstract Background A small sample size (n < 30 for each treatment group) is usually enrolled to investigate the differences in efficacy between treatments for knee osteoarthritis (OA). The objective of this study was to use simulation for comparing the power of four statistical methods for analysis of small sample size for detecting the differences in efficacy between two treatments for knee OA. Methods A total of 10,000 replicates of 5 sample sizes (n=10, 15, 20, 25, and 30 for each group) were generated based on the previous reported measures of treatment efficacy. Four statistical methods were used to compare the differences in efficacy between treatments, including the two-sample t-test (t-test), the Mann-Whitney U-test (M-W test), the Kolmogorov-Smirnov test (K-S test), and the permutation test (perm-test). Results The bias of simulated parameter means showed a decreased trend with sample size but the CV% of simulated parameter means varied with sample sizes for all parameters. For the largest sample size (n=30), the CV% could achieve a small level (<20%) for almost all parameters but the bias could not. Among the non-parametric tests for analysis of small sample size, the perm-test had the highest statistical power, and its false positive rate was not affected by sample size. However, the power of the perm-test could not achieve a high value (80%) even using the largest sample size (n=30). Conclusion The perm-test is suggested for analysis of small sample size to compare the differences in efficacy between two treatments for knee OA.

Three-year results of phase I retinal gene therapy trial for CNGA3-mutated achromatopsia: results of a non randomised controlled trial

2021 ◽  
pp. bjophthalmol-2021-319067
Author(s):  
Felix Friedrich Reichel ◽  
Stylianos Michalakis ◽  
Barbara Wilhelm ◽  
Ditta Zobor ◽  
Regine Muehlfriedel ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Primary Endpoint ◽  
Statistical Power ◽  
Small Sample Size ◽  
Controlled Trial ◽  
Statistical Significance ◽  
Small Sample ◽  
Functional Improvement ◽  
Limiting Factor ◽  
Serious Adverse Events

AimsTo determine long-term safety and efficacy outcomes of a subretinal gene therapy for CNGA3-associated achromatopsia. We present data from an open-label, nonrandomised controlled trial (NCT02610582).MethodsDetails of the study design have been previously described. Briefly, nine patients were treated in three escalating dose groups with subretinal AAV8.CNGA3 gene therapy between November 2015 and October 2016. After the first year, patients were seen on a yearly basis. Safety assessment constituted the primary endpoint. On a secondary level, multiple functional tests were carried out to determine efficacy of the therapy.ResultsNo adverse or serious adverse events deemed related to the study drug occurred after year 1. Safety of the therapy, as the primary endpoint of this trial, can, therefore, be confirmed. The functional benefits that were noted in the treated eye at year 1 were persistent throughout the following visits at years 2 and 3. While functional improvement in the treated eye reached statistical significance for some secondary endpoints, for most endpoints, this was not the case when the treated eye was compared with the untreated fellow eye.ConclusionThe results demonstrate a very good safety profile of the therapy even at the highest dose administered. The small sample size limits the statistical power of efficacy analyses. However, trial results inform on the most promising design and endpoints for future clinical trials. Such trials have to determine whether treatment of younger patients results in greater functional gains by avoiding amblyopia as a potential limiting factor.

Abstract 181: A Systematic Review of Interventions to Increase tPA Administration

Stroke ◽  
2016 ◽  
Vol 47 (suppl_1) ◽  
Author(s):  
Mollie McDermott ◽  
Lesli E Skolarus ◽  
James F Burke
Keyword(s):  
Systematic Review ◽  
Public Education ◽  
Small Sample Size ◽  
Case Reports ◽  
Meta Analysis ◽  
Experimental Studies ◽  
Fixed Effect Model ◽  
Small Sample ◽  
Eligibility Criteria ◽  
Design Elements

Introduction: Rates of tPA administration remain low nationally and globally despite its demonstrated efficacy. We performed a systematic review and meta-analysis of interventions to increase the rate of tPA administration. Methods: We searched PubMed and EMBASE to identify all studies (excluding case reports) published between 1995 and January 8, 2015 documenting interventions to increase the utilization of tPA. We screened each study with pre-specified inclusion and exclusion criteria. Design elements and study data were extracted from eligible studies. The principal summary measure was the percentage change in rate of tPA administration. Fixed and random effects meta-analytic models were built to summarize the effect of intervention compared to control as well as intervention subtypes. Results: Our search yielded 1457 results of which 25 met eligibility criteria. We identified 13 pre-post studies and 11 randomized or quasi-experimental studies. Included studies utilized EMS (n=14), telemedicine (n=6), and public education (n=5). Intervention settings included urban (n=13), rural (n=4), and combined (n=4). In a fixed effect model, tPA administration was significantly higher in the intervention arm across all studies that limited enrollment to ischemic stroke patients (n=14) with a risk ratio (RR) of 1.71. Interventions involving EMS were associated with an increased rate of tPA administration with a RR of 1.51, (95% CI: 1.43-1.59, p<0.0001); promoting public education RR = 2.62, (95% CI: 1.54-4.43, p<0.01); and utilizing telemedicine RR = 2.97, (95% CI: 2.61-3.39, p<0.0001). Conclusions: Interventions to increase tPA use appear to have considerable efficacy. Comparative inferences between intervention types are limited by small sample size and intervention heterogeneity.

A comparative assessment of orthodontic treatment outcomes using the quantitative light-induced fluorescence (QLF) method between direct bonding and indirect bonding techniques in adolescents: a single-centre, single-blind randomized controlled trial

2019 ◽  
Vol 42 (4) ◽  
pp. 441-453
Author(s):  
Aykan Onur Atilla ◽  
Taner Ozturk ◽  
Mustafa Murat Eruz ◽  
Ahmet Yagci
Keyword(s):  
Orthodontic Treatment ◽  
Small Sample Size ◽  
Controlled Trial ◽  
Small Sample ◽  
White Spot Lesion ◽  
Indirect Bonding ◽  
Signed Rank ◽  
Group 12 ◽  
Registration Number ◽  
Bonding Technique

Summary Introduction The aim of this two-arm parallel trial was to evaluate enamel demineralization in fixed orthodontic treatment using an indirect bonding technique in comparison to a direct bonding technique by the quantitative light-induced fluorescence (QLF) method. Design, settings and participants Fifty-six patients who needed fixed orthodontic treatment were randomly separated into either the direct bonding group or the indirect bonding group. The presence and extent of lesions on the buccal surfaces of all teeth, except the molar teeth, were assessed. The percentage of fluorescence loss (ΔF and ΔFmax), the degree of demineralization (ΔQ) and lesion area (WS area) were determined using the system’s software. The data were analysed with the Wilcoxon signed-rank and Mann–Whitney U-tests (P &lt; 0.05). Interventions Treatment with a direct bonding or an indirect bonding technique. Results This study was completed with 25 patients in the indirect bonding group (12 females, 13 males; mean age: 15.42 ± 1.71 years) and 26 patients in the direct bonding group (12 females, 14 males; mean age: 14.73 ± 1.65 years). In the indirect bonding technique, a few teeth (especially mandibular left canine: 50.45 ± 93.48; 95% confidence interval: −12.35 and 113.26) were found to develop significant white spot lesion (WSL) formation (P &lt; 0.05). However, the number of teeth with demineralization was higher in the direct bonding group (P &lt; 0.05). Conclusion The bonding procedures used in the indirect bonding technique contribute to reducing the degree of WSL formation. Further, the use of flowable composite adhesives in this bonding process is more effective at reducing the appearance of WSLs than in cases where conventional composite adhesives are used. Limitations The limitation of our trial may be the small sample size and the short follow-up time for the patients. Harms No harms were detected during the study. Protocol The protocol was not published before trial commencement. Registration This trial was registered post hoc at ‘Clinical Trials’ (http://www.clinicaltrials.gov), registration number (ID): NCT03738839.

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